> Preclinical therapeutic approaches in myopathic canine model
> In vivo fluorescence imaging of gene expression in muscles : where are the limits ?
> Interest of trap-free amphiphilic radicals in the treatment of mitochondrial diseases.
> Pharmacological treatment of DMD via utrophin over expression
Preclinical therapeutic approaches in myopathic canine model
After a brief introductory speech by Dr. Serge Braun and Pr. Michel Fardeau, the opening lecture on animal models and imagery was given by Dr. Stéphane Blot (National Veterinary School of Alfort, Maisons-Alfort), who discussed the use and importance of spontaneous animal pathologies to validate therapeutic strategies directed against human neuromuscular diseases. In particular, for his preclinical studies, Dr. Blot uses the golden retriever muscular dystrophy (GRMD) dog, the best animal model of Duchenne muscular dystrophy which portrays a nearly perfect replication of the human disorder. Their physiology which is similar to humans, their size and the faster evolution of their disease make them suitable tools for preclinical trials.
In vivo fluorescence imaging of gene expression in muscles : where are the limits ?
Dr. Justin Tessié (Pharmacobiology and Structural Biology Institute, Toulouse) then continued to demonstrate a non-invasive technique of in vivo fluorescence imaging of gene expression in muscles. This technique is potentially very practical in order to identify the possible early deleterious effects of a treatment or to show the improvement of histological tissue following experimentation.
Interest of trap-free amphiphilic radicals in the treatment of mitochondrial diseases.
Bernard Pucci (University of Avignon)
During the second session relating to pharmacotherapy, Dr. Bernard Pucci from the University of Avignon, discussed the interest of amphiphilic antioxidant agents in the treatment of mitochondrial diseases. Free radicals are present in a lot of natural cell processes but also in various diseases such as mitochondrial myopathies. Dr. Pucci’s preliminary findings show that in vivo and in vitro, amphiphilic antioxidant agents trap free radicals, thereby inhibiting their harmful effects in the organism. These promising results will now be extended to studies in mouse models of dysferlinopathy and Duchenne muscular dystrophy.
Pharmacological treatment of DMD via utrophin over expression.
Sabine Delaporte (CNRS, Gif-sur-Yvette)
Dr. Sabine de la Porte demonstrated that mdx mice, a model possessing histological lesions similar to Duchenne muscular dystrophy (DMD), benefit from treatments that increase utrophin, a protein which shares 80% sequence homology with dystrophin, the missing protein in DMD. Mice treated with L-arginine presented a two-threefold increase in utrophin levels resulting in a 30% reduction in necroses and a significant improvement in muscle function.
